Why the study?
Does myoblast transplantation without immunosuppression improve muscle strength and dystrophin expression in young boys with Duchenne muscular dystrophy?
Population
5 young boys with Duchenne muscular dystrophy (DMD)
Comparison
Myoblast transplantations without… vs Sham injection into the opposite biceps brachii
Design
Other, Triple blind
Follow-up
up to 18 months
Authors
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Myoblast transplantation without immunosuppression fails to improve strength in DMD; leaves open whether immunosuppression enables efficacy in future trials.
Does myoblast transplantation without immunosuppression improve muscle strength and dystrophin expression in young boys with Duchenne muscular dystrophy?
Myoblast transplantation without immunosuppression in Duchenne muscular dystrophy fails to improve muscle strength and triggers a destructive immune response, indicating that immunosuppression is required for such therapies.
Tremblay et al. (1993) studied this question.
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